iSpeak Blog

The Digital Revolution - Big Data and Real World Evidence

John J. Brennan, Ph.D.
The Digital Revolution - Big Data and Real World Evidence

Costs of pharmaceuticals to the American consumer and healthcare systems are immense, so immense in fact that Senators are calling for new actions to address the high cost of prescription drugs in America. The US spent 345 billion USD on prescription drugs in 2018 and growth estimates could reach 500 billion by 2025. A significant portion (15% to 20%) of the pharma revenue stream is used for pharmaceutical R and D, and this investment is the lifeblood of the global pharmaceutical industry. Discovery, development and commercialization of modern drug development could cost as much as 2.0 billion USD when failures and opportunity costs are taken into consideration. Establishing drug safety, determining efficacy and ensuring product quality are expensive tasks that must be monitored by sponsors and regulatory agencies, particularly in this digital health revolution.

When constructing the regulatory pathway for a New Chemical Entity (NCE) or biologic, designing and aligning the appropriate regulatory strategy for each development asset is a critical component. From Big Data, to Real World Evidence to Value Proposition and Reimbursements, regulators and sponsors must guide new product development and promotion to be consistent with product labeling as drug development and regulatory approval become more complex.

“Big Data”: The use of historical information to create a “synthetic control arm” as opposed to a placebo treatment is gaining some traction in clinical trial design. While the use of synthetic controls will never replace the randomized controlled study design, new analysis tools for complex “big data” sets could cut control groups in half or replace them altogether, especially when traditional designs become prohibitive or historical data is complete and well-characterized.

Real World Evidence (RWE) and Patient Centricity: RWE, including electronic medical records, claims and billing data, patient and disease registries and data gathered through wearable digital devices, has been little used in drug regulatory decisions regarding drug effectiveness. The industry is anticipating the release in 2020 of aFDA draft guidance on RWE and a framework for its use. Additionally, regulatory agencies are seeking information on how clinical trials can be “fit for purpose” “with the patient population prompting sponsors to investigate protocol design and interventions with patients, not just investigators, KOLs and internal experts. Patient centricity will be very relevant in future regulatory dossier reviews and approvals.

Value Proposition and Reimbursement: While regulatory approval requires demonstration of patient safety and efficacy, payer access requires a clear demonstration of a value proposition to qualify for reimbursement. Pipeline commercial development centered on pricing, market access and payer acceptance are now built into development programs long before final investment decisions are made at the governance level. Elements of the value proposition could include differentiation over standard-of-care, price, ease of use and innovative packaging, particularly as the industry shifts to a value-based reimbursement.

Strategic drug development is seeing an evolution today, one that focuses on innovation and improved patient experience while keeping a keen eye on safety and regulatory compliance.

Ensure that your firm keeps pace by ensuring your regulatory department or regulatory consultant is well-versed in FDA requirements that best position your products in today’s environment.